A case of refractory immune thrombocytopenia in which mycophenolate mofetil was effective

Abstract

We report a case in which mycophenolate mofetil (MMF) was effective in refractory immune thrombocytopenia (ITP) with bleeding tendency. An 8-year-old girl presented with purpura and epistaxis and was diagnosed with ITP. Screening for secondary ITP revealed no significant findings. For the patient’s family history, her mother has Evans syndrome and a maternal relative has thrombocytopenia. As she did not respond to immunoglobulin, corticosteroids, and steroid pulse therapy, she received thrombopoietin receptor agonists (TPO-RA) and rituximab with the approval of the Ethics Committee in our hospital; however, its effect was temporary. After MMF was introduced, the platelet count became stable and she finished the administration of corticosteroid, TPO-RA, and MMF after five, six, 14 months, respectively, from start of MMF. The combination of MMF for pediatric refractory ITP may be an effective option for stabilized platelet count.